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Haploidentical Bone Marrow Transplant for Severe Aplastic Anemia

This study is testing a new approach for people with severe aplastic anemia, a serious condition where your bone marrow doesn't make enough blood cells. It uses a bone marrow transplant from a partially matched family donor (haploidentical donor). Before the transplant, you would receive several medications: Fludarabine, Cyclophosphamide, and Rabbit ATG, along with a low dose of Total Body Irradiation (radiation). After the transplant, you would receive more Cyclophosphamide. The main goal is to see if the new bone marrow successfully grows and works for at least two years. The study plans to enroll 20 participants, aged 1 to 75 years, who have severe aplastic anemia and a suitable partially matched family donor. The study status is currently unclear.

Study design
This is an interventional study, meaning participants will receive specific treatments. It plans to enroll 20 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for two years after the transplant to check if the new bone marrow is working.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT02828592

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Recruiting
PHASE2Ages 1–75InterventionalTreatment
Northside Hospital, Inc.
~20 participants
Updated 2026-04-20 on ClinicalTrials.gov
What's tested:FludarabineCyclophosphamideTotal Body IrradiationRabbit ATG

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant
Measured over 2 years

Conditions

Severe Aplastic Anemia

Explore related trials

Aplastic Anemia

Where it's being run

2 sites across 1 states
Georgia2

Study leadership

  • Melhem Solh, MD · PRINCIPAL_INVESTIGATOR · Blood and Marrow Transplant Group of Georgia

Who to contact

Melhem Solh, MD
[email protected]
404-255-1930
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
KPS \>= 70%
Aplastic Anemia that meets the following criteria:
\<500 PMN/mm3
\<20,000 platelets
absolute reticulocyte count \<40,000/microL
markedly hypocellular (\<25% of normal cellularity)
moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above

Exclusion

poor cardiac function (LVEF \<40%)
poor pulmonary function (FEV1 \& FVC \<50% predicted)
poor liver function (bili \>= 2mg/dL)
poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
prior allogeneic transplant

What this trial measures

  • Demonstrate sustained engraftment after T-cell replete HLA-mismatched haploidentical bone marrow transplantation by collecting chimerism tests monthly following transplant2 years

    Hypothesis is that following preparative regimen and bone marrow transplantation, the 30-day graft failure rate will be \<30%.